Animal Models Core The Gene Therapy Program has access to animal facilities of importance to this P01. Dr. Wilson has managed mouse and nonhuman primate vivaria at Penn for the last 14 years. These facilities are state-ofthe- art, are operated under BSL2 and are AAALAC accredited. The University Laboratory Animal Resources (ULAR) provides clinical veterinarian support to both vivaria. The nonhuman primate facility is dedicated to Dr. Wilson's use, while the mouse vivarium was developed for the use of those faculty involved in gene therapy research. Dr. Claire Hankenson, (Assistant Professor of Laboratory Animal Medicine, Dept of Pathobiology and Senior Associate Director for Regulatory Affairs and Planning, ULAR) will serve as Director of the Core. The mouse oncogenicity study will be performed in a dedicated room within the mouse facility. All nonhuman primate experiments will be implemented by the Core with biological samples being provided to the Projects and other Cores. Lay description. A critical step in the pre-clinical development of gene therapy is the evaluation of the safety and efficacy of gene transfer in monkeys. This Core will perform these studies on behalf of the projects.